CFTR Modulators Instigate Cystic Fibrosis Market Surge, Finds Latest Report

The Cystic Fibrosis (CF) market will grow rapidly over the forecast period (2012-2019) at a Compound Annual Growth Rate (CAGR) of 30.6%. The sharp rise in market value is due to powerful new drugs entering the market, in particular the novel disease-modifying Cystic Fibrosis Transmembrane conductance Regulator (CFTR) modulators.

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Although very expensive, CFTR modulators are the first to act directly on the defective CFTR protein, which is the root cause of CF. As a result they represent the closest therapy to a cure currently available for CF patients. One CFTR modulator, Kalydeco (ivacaftor), is currently on the market, although it can only treat 5% of people with a specific mutation (O’Reilly and Elphick, 2013). Despite the low treatment population, it has already become one of the top-grossing drugs since its launch in 2012. A second CFTR modulator, lumacaftor, is expected to be launched in 2015. This drug is used in combination with Kalydeco and it is expected to be usable in over 80% of the CF population, which, combined with its large cost and uptake is expected to be the primary driver in the CF market surge.

The prevalence of CF will rise slowly over the forecast period by an estimated 10,000 cases. The primary cause of this is the increasing life expectancy of CF patients, which has rapidly grown over the past three decades as the understanding of the causes of CF has developed (Davies et al., 2007).

CF incidence remains relatively constant as it is a hereditary disease; however the mortality rate is decreasing due to improvements in treatments and disease management. Therefore, the overall mortality rate is lower than the birth rate of CF patients and so the CF population is increasing. This will eventually reach equilibrium.

The increasing prevalence is also a factor in terms of driving market growth, as many CF therapeutics must be taken indefinitely and on a regular basis.

Report Link: CysticFibrosis Therapeutics in Major Developed Markets to 2019 - CFTR ModulatorsInitiate Drive towards Personalized Treatment and Market Growth

Several key drug expirations will occur in the forecast period. The drugs set to lose their patent protection are the mucolytic Pulmozyme (dornase alfa) and the antibiotics TOBI (tobramycin) and TOBI Podhaler (tobramycin). Cheap, generic versions of TOBI and TOBI Podhaler are expected to enter the market following their patent expiration, leading to an increase in the number of antibiotics on the market and an increase in competition. The number of Pulmozyme biosimilars and the impact on pricing is expected to be limited.

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